This invention relates to gene therapy for treatment of a disease or disorder by delivering therapeutic agents to the brain. More particularly, this invention relates to gene therapy through delivery of therapeutic agents to the brain by transducing endothelial cells of blood vessels located in the brain in vivo with a vector, wherein the vector is administered intravascularly. The invention further relates to treatment of tumors by transducing gene therapy wherein endothelial cells of blood vessels of the tumor are transduced with a polynucleotide expressing a therapeutic agent.
This invention further relates to the treatment of brain tumors by gene therapy wherein endothelial cells of blood vessels located in a brain tumor are transduced with a vector which includes a polynucleotide encoding an agent which when expressed provides for inhibition, prevention or destruction of the tumor. The vector is administered intravascularly. The agent may be a negative selective marker. Upon administration of an interaction agent to the host, the growth of the brain tumor is inhibited, prevented, or destroyed, thereby leading to regression of the tumor.
Gene therapy has been used to deliver a therapeutic agent to the brain by in vivo transduction of cells which involves direct injection of a suitable vector into cells located in the brain, e.g., injection of the vector into tumor cells located in the brain.
There is a need for improved procedures for using gene therapy for delivering a therapeutic agent to the brain and in particular for the treatment of brain tumors by gene therapy which does not require direct introduction of a vector into the brain.